Tuesday, January 5, 2016

Might this open up an avenue to cure a whole host of genetic diseases and disorders?

Members of the UT Southwestern team whose research successfully halted progression of a form of muscular dystrophy in mice included (l-r) Dr. Chengzu Long, Dr. Eric Olson, Dr. Rhonda Bassel-Duby, Dr. Leonela Amoasii, John Shelton, and Alex Mireault.

If this really results in a cure for a disease — in this case Duchenne muscular dystrophy — and not just a reduction in symptoms, the breakthrough reported by Science Daily is huge news:

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. … In the study published in Science, UTSW researchers used a gene-editing approach to permanently correct the DMD mutation that causes the disease in young mice.

In 2014, Dr. Eric Olson's team first used this technique -- called CRISPR/Cas9-mediated genome editing -- to correct the mutation in the germ line of mice and prevent muscular dystrophy. This paved the way for novel genome editing-based therapeutics in DMD.

Although there are not too many cases of DMD — about 1 in 5,000 boys — there are a large number of highly debilitating genetic disorders, and I wonder if the same type of approach with a CRISPR/Cas9 system might be able to find cures for all of them.

The CRISPR genome-editing technology, which was developed by a researcher at University of California at Berkeley, was picked as the "Breakthrough of the Year" scientific development by Science.

…

"The recent groundbreaking discoveries from the Olson laboratory using genome editing to correct the genetic mutation that causes DMD have accelerated the race to find a cure for this deadly disease," said Dr. Pradeep Mammen, Associate Professor of Internal Medicine and Co-Director of the UTSW Wellstone Center. "The challenge now lies before Wellstone Center researchers to translate these discoveries in the mouse model of DMD into a therapy for patients with DMD."----------------------------UPDATE:
Only three days after I posted this information about possibly curing DMD with CRISPR/Cas9 genome editing and suggesting that it could be used in a similar way to cure other genetic disorders, it is being reported here that inherited blindness might also be cured by the same genome editing technique:A research team at the Cedars-Sinai Board of Governors Regenerative Medicine Institute focused on inherited retinitis pigmentosa, a degenerative eye disease with no known cure that can lead to blindness. The researchers used a technique known as CRISPR/Cas9 to remove a genetic mutation that causes the blindness disease. CRISPR/Cas9 is adapted from a strategy used by bacteria to fight invading viruses.